-
1
-
-
33846198829
-
A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years
-
Sifuentes M., Doroshow R., Hoft R., Mason G., Walot I., Diament M., Okazaki S., Huff K., Cox G.F., Swiedler S.J., and Kakkis E.D. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. Mol. Genet. Metab. 90 (2007) 171-180
-
(2007)
Mol. Genet. Metab.
, vol.90
, pp. 171-180
-
-
Sifuentes, M.1
Doroshow, R.2
Hoft, R.3
Mason, G.4
Walot, I.5
Diament, M.6
Okazaki, S.7
Huff, K.8
Cox, G.F.9
Swiedler, S.J.10
Kakkis, E.D.11
-
2
-
-
33747209013
-
A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)
-
Muenzer J., Wraith J.E., Beck M., Giugliani R., Harmatz P., Eng C.M., Vellodi A., Martin R., Ramaswani U., Gucsavas-Calikoglu M., Vijayaraghavan S., Wendt S., Puga A.C., Ulbrich B., Shinawi M., Cleary M., Piper D., Conway A.M., and Kemura A. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet. Med. 8 (2006) 465-473
-
(2006)
Genet. Med.
, vol.8
, pp. 465-473
-
-
Muenzer, J.1
Wraith, J.E.2
Beck, M.3
Giugliani, R.4
Harmatz, P.5
Eng, C.M.6
Vellodi, A.7
Martin, R.8
Ramaswani, U.9
Gucsavas-Calikoglu, M.10
Vijayaraghavan, S.11
Wendt, S.12
Puga, A.C.13
Ulbrich, B.14
Shinawi, M.15
Cleary, M.16
Piper, D.17
Conway, A.M.18
Kemura, A.19
-
3
-
-
46949093352
-
MPS VI Study Group. Long term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI. Final results of three clinical studies of recombinant human N-acetylgalactosamine-4-sulfatase
-
Harmatz P., Giugliani R., Schwartz I.V., Guffon N., Teles E.I., Miranda M.C., Wraith J.E., Beck M., Arash L., Scarpa M., Ketteridge D., Hopwood J.J., Piecko B., Steiner R., Whitley C.B., Kaplan P., Yu Y.F., Swiedler S.J., and Decker C. MPS VI Study Group. Long term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI. Final results of three clinical studies of recombinant human N-acetylgalactosamine-4-sulfatase. Mol. Genet. Metab. 94 (2008) 469-475
-
(2008)
Mol. Genet. Metab.
, vol.94
, pp. 469-475
-
-
Harmatz, P.1
Giugliani, R.2
Schwartz, I.V.3
Guffon, N.4
Teles, E.I.5
Miranda, M.C.6
Wraith, J.E.7
Beck, M.8
Arash, L.9
Scarpa, M.10
Ketteridge, D.11
Hopwood, J.J.12
Piecko, B.13
Steiner, R.14
Whitley, C.B.15
Kaplan, P.16
Yu, Y.F.17
Swiedler, S.J.18
Decker, C.19
-
4
-
-
38449109085
-
Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible, safe
-
Cox-Brinkman J., Timmermans R.G.M., Wijburg F.A., Donker W.E., van de Ploeg A.T., Aerts J.M.F.G., and Hollak C.E.M. Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible, safe. J. Inherit. Metab. Dis. 30 (2007) 984
-
(2007)
J. Inherit. Metab. Dis.
, vol.30
, pp. 984
-
-
Cox-Brinkman, J.1
Timmermans, R.G.M.2
Wijburg, F.A.3
Donker, W.E.4
van de Ploeg, A.T.5
Aerts, J.M.F.G.6
Hollak, C.E.M.7
-
5
-
-
57649183911
-
Home treatment with Elaprase, Naglazyme is safe in patients with mucopolysaccharidosis types II, VI, respectively
-
Bagewadi S., Roberts J., Mercer J., Jones S., Stephenson J., and Wraith J.E. Home treatment with Elaprase, Naglazyme is safe in patients with mucopolysaccharidosis types II, VI, respectively. J. Inherit. Metab. Dis. 31 (2008) 733-737
-
(2008)
J. Inherit. Metab. Dis.
, vol.31
, pp. 733-737
-
-
Bagewadi, S.1
Roberts, J.2
Mercer, J.3
Jones, S.4
Stephenson, J.5
Wraith, J.E.6
-
6
-
-
55849153560
-
Creating genetics-based infusion centers: a case study of two models
-
Laney D.J., White A.L., Rhead W.J., and Fernhoff P. Creating genetics-based infusion centers: a case study of two models. Genet. Med. 10 (2008) 626-632
-
(2008)
Genet. Med.
, vol.10
, pp. 626-632
-
-
Laney, D.J.1
White, A.L.2
Rhead, W.J.3
Fernhoff, P.4
-
7
-
-
40949141950
-
Successful management of difficult infusion-related reactions in a young patient with mucopolysaccharidosis VI receiving recombinant human arylsulfatase B (galsulfase [Naglazyme])
-
Kim K.H., Decker C., and Burton B.K. Successful management of difficult infusion-related reactions in a young patient with mucopolysaccharidosis VI receiving recombinant human arylsulfatase B (galsulfase [Naglazyme]). Pediatrics 121 (2008) e714-e717
-
(2008)
Pediatrics
, vol.121
-
-
Kim, K.H.1
Decker, C.2
Burton, B.K.3
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